Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in Study 111-202. No study drug is administered.

Approximately 200 patients will be enrolled. Patients aged 2 to 13.5 years, inclusive, on the date of consent will be enrolled. Patients aged 0 to < 2 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor. Approximately equal numbers of boys and girls will be enrolled.

Eligibility

Ages Eligible for Study:

2 Years to 13 Years (Child)

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Sampling Method:

Probability Sample

Study Population

Approximately 200 patients will be enrolled. Patients aged 2 to 13.5 years, inclusive, on the date of consent will be enrolled. Patients aged 0 to < 2 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor. Approximately equal numbers of boys and girls will be enrolled.

Criteria

Inclusion Criteria:

Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure.

Aged 2 to 13.5 years, inclusive, at study entry. Patients aged 0 to < 2 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor.

Have ACH, documented by clinical diagnosis

Are ambulatory and able to stand without assistance (not applicable for infants)

Are willing and able to perform all study procedures as physically possible.

Exclusion Criteria:

Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia)

Have any of the following disorders:

Hypothyroidism

Insulin-requiring diabetes mellitus

Autoimmune inflammatory disease

Inflammatory bowel disease

Autonomic neuropathy

Have an unstable clinical condition likely to lead to intervention during the course of the study, including progressive cervical medullary compression

Current treatment with antihypertensive medications, ACE inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, drugs known to alter renal function that is expected to continue for the duration of the study

Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time

Concomitant medication that prolongs the QT/QTc interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit

Have used any other investigational product or investigational medical device for the treatment of ACH or short stature

Have had bone-related surgery or expected to have bone-related surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.

Have any condition that, in the view of the Investigator, places the patient at high risk of poor compliance with the visit schedule or of not completing the study.

Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01603095